Oxford Drug Design announces further in vivo validation of novel oncology therapeutic mechanism
Oxford Drug Design, the Oxford-based AI drug discovery company, has announced an important milestone in the development of a potential first-in-class cancer therapy. Using its pioneering GenAI platform, the company has successfully completed in vivo validation of a novel therapeutic approach targeting multiple tumour types.
In studies using a genetically engineered mouse model that replicates the earliest mutational events in colorectal cancer, Oxford Drug Design’s lead compound demonstrated statistically significant anti-tumour activity with efficacy comparable to that of rapamycin – a benchmark therapy – while showing no detectable signs of toxicity.
A well-known limitation of rapamycin and related drugs is that tumours frequently acquire KRAS mutations that render these therapies ineffective. Oxford Drug Design’s results suggest a promising way around this obstacle. In advanced 3D tumour models derived from RAS-driven colorectal cancers, the company’s compound induced cancer cell death in settings where rapamycin failed.
These models reflect the two most aggressive human colorectal cancer subtypes (Consensus Molecular Subtypes 3 and 4) that carry KRAS mutations associated with poor clinical outcomes. The experimental work was carried out by the Cancer Research UK Scotland Institute, as part of a previously announced grant-funded collaboration.
The lead molecules arise from Oxford Drug Design’s growing portfolio of proprietary chemical scaffolds, further refined through in-house structural biology capabilities. This programme marks the company’s third first-in-class therapeutic effort to achieve clear in vivo validation using its AI-driven discovery platform.
Building on this momentum, Oxford Drug Design is already planning to extend the same differentiated capabilities into additional disease areas with significant unmet medical need.
Dr Paul Finn, CSO of Oxford Drug Design, said: “We continue to develop this breakthrough programme successfully against major tumours, applying our integrated expertise in generative AI and target biology. A significant milestone has been achieved and our rapidly advancing efforts are now focused on bringing this potential first-in-class treatment into the clinic”.
Professor Sarah Blagden, FRCP, PhD, Oxford University Professor of Experimental Oncology, Lead of the Oxford Cancer Trials Office and Member of the Oxford Drug Design Scientific Advisory Board, added: “These latest results of Oxford Drug Design are impressive. The relative strength of the lead candidate over rapamycin in mutated colorectal cancers points to broad clinical potential for this exciting, novel therapy. I look forward to further progress as it approaches human trials”
Dr Alan D. Roth, CEO of Oxford Drug Design, commented: “Together with our CRUK Scotland Institute collaborators, our progress in this innovative approach against major tumours continues to validate the accuracy of our dual discovery platform, now poised to deliver superiority over established treatments in oncology. We continue our efforts to benefit patients with this breakthrough programme while building our pipeline with further first-in-class treatments.”
Professor Owen Sansom, FRSE, FMedSci, Director of the CRUK Scotland Institute (previously known as CRUK Beatson Institute), Scientific Director of the CRUK Scotland Centre and Director of the National Mouse Genetic Network, UK, said: “The collaboration – led by Dr. Valeria Pavet (Head of the Translational Research Collaborations Unit at CRUK SI) and Dr. Andrew Campbell (Research Team Lead, CRUKSI) – with Oxford Drug Design is an excellent example of the synergistic benefits arising from combining excellent academic and commercial expertise to advance a novel therapeutic approach. We look forward to continuing working together as the project advances towards the clinic.”